Systems Pharmacology Approach to Uncontrolled Pediatric Asthma

Pediatric Asthma Clinical Trial

Official title:

Systems Pharmacology Approach to Uncontrolled Pediatric Asthma (SysPharmPediA)

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT04865575
• Other study ID #: NL55788.041.15
• Status: Completed
• Start date: January 12, 2018
• Est. completion date: October 31, 2020

Study information

• Verified date: April 2021
• Source: Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA)
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial Summary

Background:

Asthma is a heterogeneous respiratory disease and the most common chronic disease in children. A small subset of children has continuous poor asthma control despite appropriate adherence to asthma medication. There is a clinical need to identify these children as early as possible to optimize treatment and/or to find therapeutic alternatives. Therefore, the "Systems Pharmacology approach to uncontrolled Pediatric Asthma" (SysPharmPediA) study was set up.

Objective:

To establish a cohort of pediatric moderate-to-severe uncontrolled and controlled patients with asthma in order to investigate pathophysiological mechanisms underlying uncontrolled moderate-to-severe asthma in children on maintenance treatment, using a multi-omics systems medicine approach.

Methods:

In this multicenter observational case-control study, moderate-to-severe asthmatic children (n=145, age 6-17 years), were included in specialized hospitals in four European countries (Netherlands, Germany, Spain and Slovenia). Recruited subjects were selected based on good asthma control (controlled asthmatics, n=54) or poor asthma control / recurrent exacerbations (uncontrolled asthmatics, n=91). Comprehensive details concerning demographics, current and past patient/family history and clinical characteristics were collected. In addition, systems-wide omics layers, including epi(genomics), transcriptomics, microbiome, proteomics and metabolomics will be evaluated from multiple collected, relatively non-invasive, samples of from the recruited individuals, such as: blood, feces, saliva, nasal swabs and exhaled breath. Follow-up visits were performed 6 and 12 months after inclusion.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 145
• Est. completion date: October 31, 2020
• Est. primary completion date: October 31, 2019
• Accepts healthy volunteers: No
• Gender: All
• Age group: 6 Years to 18 Years

Eligibility

Inclusion Criteria:

General:

• Between 6-17 years of age

• Doctor diagnosis of asthma

In addition to the general criteria, a case with uncontrolled asthma (case group A) should meet:

• Asthma treatment Global INitiative of Asthma (GINA) step 3 or higher

= 1of the following criteria (based on the guideline 'Childhood Asthma' of the section paediatric pulmonology of the Dutch Society of Paediatricians, 2007):

• Frequent exacerbations requiring OCS use (=1 in the past year) and/or

• Severe exacerbations requiring hospitalization or ER visits in the past year and/or

• ACQ/ACT scores indicating uncontrolled asthma

In addition to the general criteria, a case with an acute exacerbation (case group B) should meet:

• Asthma treatment Global INitiative of Asthma (GINA) step 2 or higher

• Current severe asthma exacerbation requiring hospitalization

In addition to the general inclusion criteria, a control (controlled asthma) should meet the following criteria:

• Asthma treatment Global INitiative of Asthma (GINA) step 3 or higher

• Lack of severe exacerbations requiring OCS use or hospitalizations or ER visits in the past year

• ACQ/ACT scores indicating well controlled asthma or few reported asthma symptoms during asthma checks in the past year.

Exclusion Criteria:

• Recent use of a course of antibiotics (< 1 month). This will affect the microbiome analyses.

A patient can be re-screened 1 month after the use of the antibiotic treatment has finished.

Related Conditions & MeSH terms

• Asthma
• Pediatric Asthma
• Uncontrolled Asthma

Locations

Country	Name	City	State
Germany	University Regensburg	Regensburg
Netherlands	Utrecht University	Utrecht
Slovenia	University of Maribor	Maribor
Spain	University of the Basque Country	San Sebastián
Spain	Universidad de La Laguna	Santa Cruz De Tenerife
Sweden	Karolinska University Hospital	Stockholm

Sponsors (10)

Lead Sponsor

Collaborator

Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA)

Hospital Universitario Donostia, San Sebastián, Spain., Instituto de Salud Carlos III, Madrid, Comunidad de Madrid, Spain., Karolinska Institutet, Karolinska Institutet, Stockholm, Sweden, Universidad de La Laguna, San Cristóbal de La Laguna, Santa Cruz de Tenerife, Spain., University Children's Hospital, University of Regensburg, Regensburg, Germany, University of Maribor, Maribor, Slovenia., University of the Basque Country (UPV/EHU), San Sebastián, Spain., Utrecht University, Utrecht, the Netherlands.

Countries where clinical trial is conducted

Germany,  Netherlands,  Slovenia,  Spain,  Sweden, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Other	Age	Age in years	Baseline (+ follow-up at 6 and 12 months after baseline inclusion)
Other	Sex	Female/Male	Baseline (+ follow-up at 6 and 12 months after baseline inclusion)
Other	BMI	kg/m2	Baseline (+ follow-up at 6 and 12 months after baseline inclusion)
Other	Uncontrolled asthma	according to the Asthma Control Test (ACT) questionnaire (<19 score) and frequent exacerbation within the past 12 months (>n of exacerbation in the past 12 months	Baseline (+ follow-up at 6 and 12 months after baseline inclusion)
Other	Exacerbations	Exacerbation requiring OCS use, hospitalization or ER visit	Baseline (+ follow-up at 6 and 12 months after baseline inclusion)
Other	Spirometry	FEV1 (L, percent predicted value)	Baseline
Other	Pediatric Asthma Quality of Life Questionnaire (PAQLQ)	Questionnaire including 23 questions in 3 domains (symptoms, activity limitation, emotional function) and a 7-point scale (7 = not bothered at all; 1 = extremely bothered)	Baseline (+ follow-up at 6 and 12 months after baseline inclusion)
Other	Atopy	Questionnaire on history of allergic diseases	Baseline (+ follow-up at 6 and 12 months after baseline inclusion)
Other	Current asthma medication intake	ICS: inhaled corticosteroids, LTRA: leukotriene antagonist, SABA: short-acting beta agonist, LABA: long-acting beta agonist, OCS: oral corticosteroids, Omalizumab, Mepolizumab	Baseline (+ follow-up at 6 and 12 months after baseline inclusion)
Other	Asthma severity	Asthma Control Test (ACT)	Baseline (+ follow-up at 6 and 12 months after baseline inclusion)
Primary	Saliva and stool microbiome	Differences in 16S rRNA microbial a- and ß- diversities between cases and controls	Baseline
Primary	Exhaled breath metabolome	Differences in presence and levels of volatile organic compounds between cases and controls	Baseline